BACKGROUND AND CURRENT LANDSCAPE Since the first clinical trial in humans (1989), there have been approximately 2,500 registered clinical trials in gene therapy. Considered as a disruptive new technology, gene therapies provide a radical new approach that targets the cause of the illness rather than the symptoms, and have potential in a number of therapeutic areas. THE ISSUE Gene therapies have the benefit of potentially producing significant cost offsets by replacing long-term treatments, reducing in-patient care / hospitalizations and further exacerbations or adverse events. The caveat, however, is the elevated upfront costs that gene therapies incur. APPROACH Through a targeted review of current literature (e.g. PubMed) and databases (e.g. Datamonitor/EMA), this paper aims to map the current gene therapy landscape (i.e. current pipeline products and developing policies) and highlight the evolving hurdles and solutions of access for gene therapies. The final goal is to encourage a debate on the potential impact that gene therapy could have on the pharmaceutical industry and the healthcare systems/budgets. THE SOLUTION Alternative financing models should consider the following features and benefits that gene therapy can offer:

• Development of innovative fee-for-service payer models to potentially high upfront-costs leading to more cooperative and combined stakeholder approaches that may transcend sectors
• Transition from symptom management to cure (e.g. impact on chronic diseases)
• Impact on in-patient pathways and care
• Long-term potential to shift treatment costs from traditional treatments
• Possibility to cure previously untreatable/incurable diseases (e.g. genetic mutations, viruses, oncology, etc.)

IMPACT Payers and policymakers must consider more innovative and collaborative solutions in any future financial models to facilitate access of valuable new therapies to treat diseases with high burden and significant unmet needs, whilst balancing ever-restricted healthcare budgets. This paper intends to explore the potential options available along with pros and cons for each.